On July 11, 2018 the US Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) offered 6 new draft guidance documents on human gene therapies. The documents offer a look at what it expects from an industry that is becoming very popular, reported RAPS.
The guidance documents focus on developing hemophilia, rare disease and retinal disorder gene therapies, chemistry, manufacturing and control (CMC) information, long term follow-up observational studies collecting data on adverse events, and lastly one on the testing of retroviral vector-based therapies.
In December 2017, FDA approve a new gene therapy from Spark Therapeutics for the treatment of children and adult patients with an inherited form of vision loss that could result in blindness, it was priced at $850,000. Additionally, last November, FDA announced its policy framework for regenerative medicine.
Hemophilia
The draft guidance for hemophilia is 15 pages long and provides recommendations on the clinical trial design and development of coagulation factor VIII and IX. The draft guidance also includes advice on preclinical and clinical concerns to support the development of hemophilia gene therapies (GTs).1
Rare Disease
The rare disease draft guidance is 14 pages long and mostly provides recommendations on “developing a GT product intended to treat a rare disease in adult and/or pediatric patients.”1However, the guidance related to the manufacturing, preclinical, and clinical trial design issues for all phases. The ultimate goal of the FDA is to assist the sponsors designing clinical development programs.
Retinal Disorders
The 13-page draft guidance is geared towards stakeholders developing GTs for retinal disorders. The focus of the document provides recommendations related to product development, preclinical testing and clinical trial design.1
CMC
The CMC draft guidance is 54 pages long and provides sponsors of a GT investigational new drug application (IND) with recommendations to assure product safety, identity, quality, purity, and strength of the investigational product. FDA acknowledges that the field of gene therapy has progressed rapidly and therefore provides justification for a longer draft guidance document.
Long Term Follow-Up
The 36-page draft guidance provides recommendations relation to the design of protocols for long term follow-up observational studies. The draft, once finalized, will supersede the document titled “Guidance for Industry: Gene Therapy Clinical Trials–Observing Participants for Delayed Adverse Events.”
Testing of Retroviral Vector-Based Human Gene Therapies
This 16-page draft guidance is aimed to provide sponsors of retroviral vector-based human GTs with advice regarding the testing for replication competent retrovirus (RCR).
FDA said, “Recommendations include the identification and amount of material to be tested, and general testing methods,” as well as, “In addition, recommendations are provided on monitoring patients for evidence of retroviral infection after administration of retroviral vector-based gene therapy products.”
Pearl Pathways delivers seasoned, responsive consultants who can design global regulatory strategies to trim months and dollars off the product development path or expertly negotiate global health authorities’ interactions and filings. Learn more about our team.